I was also diagnosed in 2012 and has surgery in 2013. I continue on meds and have many health issues still. I haven’t had a full recovery and dramatic weight loss or anything. I’m now starting to regret surgery as it has left me very limited in what I can do. I was far more mobile before. I miss my life. I’ve lost everything since having surgery!

I, too, was diagnosed with a pituitary tumor and subsequently had surgery to remove it. I still struggle day to day with many issues…word finding, comprehension, emotions. I also struggle with finding Cushings disease support groups in our area.

A simple test that measures free cortisol levels in saliva at midnight — called a midnight salivary cortisol test — showed good diagnostic performance for Cushing’s syndrome among a Chinese population, according to a recent study. The test was better than the standard urine free cortisol levels and may be an alternative for people with end-stage kidney disea […]

Your case is every similar to mine. I wasn’t a dancer but I did play multiple sports in high school and played college basketball. I saw doctor Yuen at Swedish as well and many more doctors as well. I have never got my case or my symptoms solved. Over 4 years of doctors and testing. They found I had a pituitary tumor and mildly high cortisol in my 24 hour Ur […]

Thanks for sharing your story. In February it will be 6 years since I’ve had my pituitary surgery. My health is constantly up and down as well. I was just wondering if you’re treated for depression or anxiety at all? Also, have you found any exercises or physical therapy to be helpful?

Jill wrote: 'In December 2004 my dad who had addison's for over 30 years had a triple bypass surgery 6 days before Christmas. The surgery was an amazine success and it was predicted he would be home before Christmas. Day 2 following surgery the hospital neglected to give him his steriods for his Addison's for 22 hours, which they were complete […]

A man with Cushing’s disease — caused by an adrenocorticotrophic hormone (ACTH)-secreting pituitary adenoma — who later developed metastases in the central nervous system without Cushing’s recurrence, was successfully treated over eight years with radiation and chemotherapy, according to a case report.

Meta

Relacorilant, an investigational therapy developed by Corcept Therapeutics, may effectively manage the effects of excess cortisol in patients with Cushing’s syndrome, interim data from an ongoing Phase 2 trial show.

In particular, the treatment significantly improved sugar tolerance and the levels of osteocalcin, a bone growth biomarker commonly suppressed by excess cortisol.

In addition to the early efficacy data, the study showed that the treatment was generally safe and well-tolerated by the patients, with adverse events reportedly mild in severity.

These findings supported the launch of the Phase 2 trial in patients with Cushing’s syndrome. In the trial, roughly 30 patients are receiving escalating doses of relacorilant for a total of 12 weeks.

Patients were divided into two groups. The first group, which includes 17 patients, receives the lowest dose — 100 mg/day of relacorilant for four weeks, followed by 150 mg/day for four weeks, and then 200 mg/day for the last four weeks. The second group, called the high-dose cohort, is treated with a similar regimen but with a starting dose of 250 mg/day and a final dose of 350 mg/day.

Patients in the low-dose group had a significant improvement in their glucose tolerance and a 60% increase in blood osteocalcin.

In addition, the treatment reduced the blood pressure in 45% of patients with uncontrolled high blood pressure from cortisol excess. Importantly, the results after 12 weeks of relacorilant were similar to those seen after six months of Korlym treatment.

Safety data continues to show a positive profile, with no evidence of serious adverse effects and no affinity toward the progesterone receptor, which is a major drawback of Korlym.

“Relacorilant’s clinical results are striking because the doses these patients received were the study’s lowest. We did not expect patients to experience any meaningful clinical benefit, but they clearly did,” Robert S. Fishman, MD, chief medical officer of Corcept, said in the release. “We look forward to presenting data from these low-dose patients at the AACE meeting next week. With the trial’s final, high-dose cohort fully enrolled, we will have final data in the third quarter.”

Supported by these preliminary data, Corcept has accelerated the preparations for a Phase 3 trial on relacorilant in Cushing’s syndrome patients.

PHILADELPHIA, April 04, 2018 — SteroTherapeutics, a privately held biopharmaceutical company developing therapies focused on metabolic diseases including non-alcoholic steatohepatitis (NASH), announced today that the U.S. Food and Drug Administration has granted orphan drug designation for ST-002 in the treatment of nonalcoholic fatty liver disease, nonalcoholic steatosis and hyperglycemia in patients with Cushing’s syndrome.

“We are pursuing a drug that has a very real potential to become the optimal agent of choice and a standard of care for these Cushing’s patients,” said Manohar Katakam Ph. D., CEO of SteroTherapeutics. “Our clinical trial will target multiple critical metabolic-related outcomes including the reduction of triglycerides, insulin resistance, weight loss, and the prevention and/or abrogation of hepatic steatosis and fibrosis.”

“The FDA’s orphan-drug designation for Fluasterone highlights the significant unmet and underserved needs for treatment in these individuals,” added Dr. Katakam. “We look forward to realizing the benefits and promise of this potential for Fluasterone in Cushing’s syndrome patients.”

The Orphan Drug Act became law in 1983. Fewer than 5,000 applicants have received this designation, according to the FDA website. Rare conditions are often described as orphan diseases or disorders when there are few or no treatment options. There are approximately 7,000 known orphan diseases.

The FDA’s Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.

The designation allows the sponsor of the drug to be eligible for various incentives, including a seven-year period of U.S. marketing exclusivity upon regulatory approval of the drug, as well as tax credits for clinical research costs, annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

Cushing syndrome occurs when a patient’s body is exposed to high levels of the hormone cortisol over a long period of time (chronic hypercortisolemia) . Cushing syndrome, sometimes called hypercortisolism, affects 15,000 to 20,000 patients in the United States.

Too much cortisol can produce some of the hallmark signs of Cushing syndrome — a fatty hump between a patient’s shoulders, a rounded face, and pink or purple stretch marks on the skin. Cushing syndrome can also result in high blood pressure, bone loss and upper body obesity, increased fat around the neck, and relatively slender arms and legs. Diabetes is frequently a complication found in Cushing’s syndrome patients. These patients also develop nonalcoholic fatty disease and steatosis as a result of the chronic hypercortisolism.

About SteroTherapeutics

SteroTherapeutics, a Philadelphia, PA area based company, is focused on developing novel therapies for significant unmet needs in metabolic disease including liver diseases.

SteroTherapeutics lead products have been proven in previous human studies to possess a strong safety profile and established mechanisms of action. The company’s strategic intent is to focus on understanding disease pathways and how to safely treat and restore an optimal quality of life. SteroTherapeutics is managed by a veteran team that has significant experience in the pharmaceutical and biotechnology industry. The team has specific experiences in the development, manufacturing and commercialization of small molecule and biologics based products.

Valassi E, et al. – This study was performed to assess how frequently preoperative medical treatment (PMT) was given to Cushing’s syndrome (CS) patients across Europe and to investigate differences in preoperative characteristics of patients who receive PMT and those who undergo primary surgery. In addition, the physicians determined if PMT influenced the postoperative outcome in pituitary-dependent CS (PIT-CS). In contrast with adrenal-dependent CS (ADR-CS), CS from an ectopic source (ECT-CS) and PIT-CS exhibited greater likelihood of receiving PMT. Data reported more severe clinical features at the diagnosis and poorer quality of life in PIT-CS patients treated with PMT. The interpretation of immediate postoperative outcome could be confounded with PMT. They recommended follow-up to definitely evaluate surgical results.

Methods

A total of 1,143 CS patients entered into the ERCUSYN database from 57 centres in 26 countries.

During this study, 69% patients presented with PIT-CS, 25% adrenal-dependent CS (ADR-CS), 5% CS from an ectopic source (ECT-CS), and 1% were classified as having CS from other causes (OTH-CS).

Results

In this study, 20% of patients took PMT.

PMT was offered more frequently in ECT-CS and PIT-CS compared to ADR-CS (p < 0.001).

Ketoconazole (62%), metyrapone (16%), and a combination of both (12%) were the most commonly used drugs.

The median (interquartile range) duration of PMT was 109 (98) days.

More severe clinical features at diagnosis and poorer quality of life were noted in PIT-CS patients treated with PMT compared to those undergoing primary surgery (SX) (p < 0.05).

PIT-CS patients treated with PMT were more likely to have normal cortisol (p < 0.01) and a lower remission rate (p < 0.01) within 7 days of surgery.

Between SX and PMT groups, no differences in morbidity or remission rates were observed within 6 months of surgery.

People with high cortisol levels have lower muscle mass and higher visceral fat deposits, putting them at a greater risk for cardiovascular disease, new research shows.

High levels of cortisol can result from a variety of reasons, including Cushing’s disease and adrenal tumors. Most adrenal tumors are found to be non-functioning, meaning they do not produce excess hormones. However, up to 47 percent of patients have mild autonomous cortisol excess (MACE).

Long-term studies have shown that as a group, patients with MACE tend to have increased cardiovascular risk factors, such as hypertension, type 2 diabetes mellitus (DM2), obesity, and high lipid levels, which are associated with higher cardiovascular death rates.

Abdominal adiposity, which refers to fat deposits around the abdomen and stomach, and central sarcopenia, referring to loss of skeletal muscle mass, are both known to be linked to higher cardiovascular risk and increased mortality.

Overt hypercortisolism is known to lead to increased visceral adiposity (body fat stored within the abdominal cavity) and muscle loss. However, little is known about the body composition of patients with adrenal adenomas and MACE.

Therefore, researchers set out to determine whether central sarcopenia and adiposity are present in patients with MACE, and whether they can be markers of disease severity in patients with adrenal adenomas. To determine this, researchers used body composition measurements of 25 patients with Cushing’s disease, 48 patients with MACE, and 32 patients with non-functioning adrenal tumors (NFAT) using abdominal CTs.

Specifically, researchers looked at visceral fat, subcutaneous fat, and total abdominal muscle mass. Visceral fat refers to fat around organs, and it is “deeper” than subcutaneous fat, which is closer to the skin.

Results showed that, compared to patients with non-functional tumors, those with Cushing’s disease had a higher visceral to total (V/T) fat ratio but a lower visceral to subcutaneous (V/S) fat ratio. In MACE patients, however, both ratios were decreased compared to patients with non-functional tumors.

Cushing’s disease patients also had 10 cm2 less total muscle mass, compared to patients with non-functional tumors.

An overnight dexamethasone suppression test was conducted in these patients to determine levels of cortisol in the blood. The next morning, cortisol levels were checked. High levels of cortisol indicate the presence of a disease, such as MACE or Cushing’s disease.

After administering the test, researchers determined that for an increase in cortisol in the morning, there was a correlating increase in the V/T ratio and the V/S fat ratio, and a decrease in the mean total muscle mass.

Therefore, the higher the degree of hypercortisolism, the lower the muscle mass and the higher the visceral adiposity.

These results could prove to be clinically useful as both visceral adiposity and low muscle mass are risk factors of a number of diseases, including cardiovascular disease.

“Body composition measurement may provide an additive value in making a diagnosis of clinically important MACE and aid in individualizing management of patients with ACAs and MACE,” the researchers concluded.

The study’s senior author was Dr. Prashant Chittiboina, MD, from the Department of Neurosurgery, Wexner Medical Center, The Ohio State University, in Columbus, Ohio.

Microadenomas – tumors in the pituitary gland measuring less than 10 mm in diameter – that release corticotropin, or corticotropinomas, can lead to Cushing’s disease. The presurgical detection of these microadenomas could improve surgical outcomes in patients with Cushing’s.

Stimulation with corticotropin-releasing hormone (CRH), which increases glucose uptake, has been suggested as a method of increasing the detection of adenomas with 18F-FDG PET, by augmenting the uptake of 18F-FDG – a glucose analog.

However, previous studies aiming to validate this idea have failed, leading the research team to hypothesize that it may be due to a delayed elevation in glucose uptake in corticotropinomas.

The scientists used clinical data to determine the effectiveness of CRH in improving the detection of corticotropinomas with 18F-FDG PET in Cushing’s disease.

They found that CRH increased glucose uptake in human and mouse tumor cells, but not in healthy mouse or human pituitary cells that produce the adrenocorticotropic hormone (ACTH). Exposure to CRH increased glucose uptake in mouse tumor cells, with a maximal effect at four hours after stimulation.

Similarly, the glucose transporter GLUT1, which is located at the cell membrane, was increased two hours after stimulation, as was GLUT1-mediated glucose transport.

Overall, the study shows that corticotropin-releasing hormone (CRH) leads to a specific and delayed increase in glucose uptake in tumor corticotrophs.

“Taken together, these novel findings support the potential use of delayed 18F-FDG PET imaging following CRH stimulation to improve microadenoma detection in [Cushing’s disease],” researchers wrote. The scientists are now conducting a clinical trial to further explore this promising finding.